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Journal of Allergy and Clinical Immunology, The, ISSN 0091-6749, 2015, Volume 136, Issue 3, pp. 692 - 702.e2
Background Wiskott-Aldrich syndrome (WAS) is a severe X-linked immunodeficiency characterized by microthrombocytopenia, eczema, recurrent infections, and... 
Allergy and Immunology | Wiskott-Aldrich syndrome | primary immunodeficiency | B cell | gene therapy | lentiviral vector | RHEUMATOID-ARTHRITIS | SYSTEMIC-LUPUS-ERYTHEMATOSUS | COMPLEMENT | ADHERENCE TEST | ACTIN POLYMERIZATION | SYNDROME PROTEIN | IMMUNOLOGY | TRANSPLANTATION | PRIMARY IMMUNODEFICIENCY DISEASES | ALLERGY | ACTIVATING FACTOR | LYMPHOCYTES | Wiskott-Aldrich Syndrome Protein - genetics | Bone Marrow - immunology | Humans | Recombinant Fusion Proteins - therapeutic use | Child, Preschool | Infant | Male | Transplantation, Autologous | Gene Expression Profiling | Autoantibodies - biosynthesis | Wiskott-Aldrich Syndrome - genetics | Wiskott-Aldrich Syndrome - therapy | Hematopoietic Stem Cells - immunology | Bone Marrow - metabolism | B-Lymphocyte Subsets - immunology | Lentivirus - genetics | Wiskott-Aldrich Syndrome - pathology | Child | Immunoglobulins - biosynthesis | Gene Expression | Wiskott-Aldrich Syndrome - immunology | Transduction, Genetic | B-Lymphocyte Subsets - pathology | Hematopoietic Stem Cell Transplantation | Immunophenotyping | Hematopoietic Stem Cells - metabolism | B-Cell Activating Factor - metabolism | B-Lymphocyte Subsets - metabolism | Wiskott-Aldrich Syndrome Protein - immunology | B-Cell Activating Factor - genetics | Bone Marrow - pathology | Transplantation Conditioning | Genetic Vectors | Genetic Therapy - methods | Autoimmunity | Care and treatment | Genetic vectors | Genes | Immunodeficiency | Genetic research | Lymphomas | Transplantation | Gene therapy | Health aspects | Hematopoietic stem cells | Bone marrow | Immunoglobulins | Patients | Immune Deficiencies, Infection, and Systemic Immune Disorders | WASp, Wiskott-Aldrich syndrome protein | WAS, Wiskott-Aldrich syndrome | GT, Gene therapy | PB, Peripheral blood | IVIg, Intravenous immunoglobulin | VCN, Vector copy number | HSC, Hematopoietic stem cell | HD, Healthy donor | BAFF, B cell–activating factor | BM, Bone marrow | SDF-1α, Stromal cell–derived factor 1α
Journal Article
Journal Article
Journal of Allergy and Clinical Immunology, The, ISSN 0091-6749, 2014, Volume 133, Issue 3, pp. 799 - 806.e10
Journal Article
Journal of Allergy and Clinical Immunology, The, ISSN 0091-6749, 2011, Volume 127, Issue 6, pp. 1417 - 1419.e3
Journal Article
Journal of Allergy and Clinical Immunology, The, ISSN 0091-6749, 2011, Volume 127, Issue 6, pp. 1368 - 1375.e8
Journal Article
by Schmidt, Manfred and Verhoeyen, Els and Gobbo, Emilie and Divers, Dominique and Oudrhiri, Noufissa and Griscelli, Frank and Bennaceur-Griscelli, Annelise and Klatzmann, David and Anguela, Xavier M and Sharma, Rajiv and Sharma, Rajiv and Li, Hojun and Li, Hojun and Haurigot, Virginia and Haurigot, Virginia and Haurigot, Virginia and Bhagwat, Anand and Bhagwat, Anand and Davidson, Robert and Davidson, Robert and Zhou, Shangzhen and Zhou, Shangzhen and Zhou, Shangzhen and Doyon, Yannick and Gregory, Philip D and Gregory, Philip D and Gregory, Philip D and Holmes, Michael C and Holmes, Michael C and Holmes, Michael C and High, Katherine A and Carbonaro, Denise and Shaw, Kit and Jin, Xiangyang and Geiger, Sabine and Mishra, Suparna and Cooper, Aaron and DeOliveira, Satiro and Sokolic, Rob and Candotti, Fabio and Carmo, Marlene and Arumugam, Paritha and Alonso-Ferrero, Maria and Schambach, Axel and Schambach, Axel and Baum, Christopher and Baum, Christopher and Baum, Christopher and Risma, Kimberly and Malik, Punam and Jordan, Michael and Rivat, Christine and Booth, Claire and Thrasher, Adrian and Whilding, Lynsey and Archibald, Kyra and Oberg, Daniel and Golan, Talia and Hubert, Ayala and Shemi, Amotz and Khvalevsky, Elina Zorde and Gabai-Malka, Racheli and Focht, Gili and Brunschwig, Zivia and Raskin, Stephen and Goldberg, Nahum and Ben-David, Eli and Peretz, Tamar and Eliakim, Rami and Dankur, Alan and Galun and Rachmur, Itzik and Domb, Avi and Kopelman, Yael and Hantz, Yael and Lahav, Mor and Arbel-Alon, Sagit and Dickson, George and Barkats, Martine and Daboussi, Fayza and Silva, Georges and Cedrone, Frederic and Epinat, Jean Charles and Juillerat, Alexandre and Valton, Julien and Montini, Eugenio and Biffi, Alessandra and Biffi, Alessandra and Calabria, Andrea and Calabria, Andrea and Biasco, Luca and Biasco, Luca and Cesani, Martina and Cesani, Martina and Benedicenti, Fabrizio and Benedicenti, Fabrizio and Plati, Tiziana and Leo, Simone and Zanetti, Gianluigi and Aiuti, Alessandro and ...
Human Gene Therapy, ISSN 1043-0342, 10/2012, Volume 23, Issue 10, pp. A1 - A173
Journal Article
The Journal of Allergy and Clinical Immunology, ISSN 0091-6749, 09/2019, Volume 144, Issue 3, pp. 825 - 838
Thrombocytopenia is a serious issue for all patients with classical Wiskott-Aldrich syndrome (WAS) and X-linked thrombocytopenia (XLT) because it causes severe... 
Wiskott-Aldrich syndrome | X-linked thrombocytopenia | gene therapy | platelets | CLEARANCE | CD40 LIGAND | CELL TRANSPLANTATION | SYNDROME PROTEIN | NEUTROPHILS | SPLENECTOMY | IMMUNOLOGY | ACTIVATED PLATELETS | ALLERGY | MUTATIONS | EXPRESSION | Genetic research | Care and treatment | Genetic aspects | Gene therapy | Analysis | Flow cytometry | Pediatrics | Transplants & implants | Medical services | Clinical trials | Agglomeration | Coexistence | Bleeding | Defects | Proteins | Genotype & phenotype | Signal transduction | Protein composition | Restoration | Blood platelets | Ultrastructure | Thrombocytopenia | Phenotypes | Immune reconstitution | Electron microscopy | Patients | Microscopy | Protein expression | Software | Mutation | Platelets | FERMT3, Fermitin family homolog 3 | TEM, Transmission electron microscopy | WAS, Wiskott-Aldrich syndrome | HMGB1, High-mobility group box 1 | δ-g, Electron-dense granule | GPX1, Glutathione peroxidase 1 | FU, Follow-up | sCD62P, Soluble P-selectin | sCD40L, Soluble CD40 ligand | BAFF, B cell–activating factor | CD62P, P-selectin | HSCT, Hematopoietic stem cell transplantation | WASp, Wiskott-Aldrich syndrome protein | MFI, Mean fluorescence intensity | LV, Lentivirus | CT, Closure time | GT, Gene therapy | OCS, Open canalicular system | PRP, Platelet-rich plasma | vWF, von Willebrand factor | STAT3, Signal transducer and activator of transcription 3 | HD, Healthy donor | ADP, Adenosine diphosphate | ROS, Reactive oxygen species | XLT, X-linked thrombocytopenia
Journal Article
The Journal of Allergy and Clinical Immunology, ISSN 0091-6749, 05/2019, Volume 143, Issue 5, pp. 1952 - 1956.e6
Journal Article
1991, Inscriptiones Christianae Italiae. Subsidia, ISBN 9788872280775, Volume 3, xiv, 382
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